Biotech and/ or pharma companies are developing drugs that could potentially benefit VWM patients. Before those drugs can become available for VWM patients, they need to be tested in clinical trials. However, as VWM can have an unpredictable disease course & phenotype, clinical trial design in VWM is challenging. Furthermore, since VWM is a rare disease, there is a low number of patients available for such trials.
To address these challenges, the VWM Consortium and representatives of the VWM patient advocates developed a ‘core clinical trial protocol’. This describes our optimal clinical trial design for VWM. Next to that, the ‘core protocol’ proposes how sharing of control/ placebo data can facilitate multiple trials in a small patient population, thereby limiting the number of patients on placebo during such trials. This ‘core protocol’ is aimed to function as a template for future VWM trials to collect safety, tolerability, and efficacy data for treatment assessment and marketing authorization. Those trials need to be organised and sponsored by the biotech and/ or pharma companies that develop potentially beneficial drugs for VWM patients.
A pre-print of this paper (not yet peer-reviewed or officially published in a scientific journal) can be read here